Table 1.
Patient demographics
| Idebenone 900 mg/day (n = 55)a | Placebo (n = 30)a | Total (n = 85)a | |
|---|---|---|---|
| Age, mean ± SD; [median] (range) (years) | 33.8 ± 14.8; [30.0] (14–63) | 33.6 ± 14.6; [28.5] (14–66) | 33.7 ± 14.6; [30.0] (14–66) |
| Sex | |||
| Male, n (%) | 47 (85.5) | 26 (86.7) | 73 (85.9) |
| Female, n (%) | 8 (14.5) | 4 (13.3) | 12 (14.1) |
| Mutations, n (%) | |||
| m.11778 G>A | 37 (67.3) | 20 (66.7) | 57 (67.1) |
| m. 14484 T>C | 11 (20) | 6 (20) | 17 (20.0) |
| m. 3460 G>A | 7 (12.7) | 4 (13.3) | 11 (12.9) |
| BMI, mean ± SD; [median] (range) (kg/m2) | 24.2 ± 4.4; [23.5] (16.1–37.0) | 24.9 ± 4.4; [24.5] (18.9–35.1) | 24.5 ± 4.4; [23.6] (16.1–37.0) |
| Months since onset of vision loss, mean ± SD; [median] (range) | 22.8 ± 16.2; [17.8] (3–62) | 23.7 ± 16.4; [19.2] (2–57) | 23.1 ± 16.2; [18.2] (2–62) |
| Patients with onset of symptoms >1 year, n (%) | 36 (65.5) | 19 (63.3) | 55 (64.7) |
| Patients with logMAR ≥ 1.0,bn (%) one eye/both eyes | 5 (9.4)/45 (84.9) | 2 (6.9)/25 (86.2) | 7 (8.5)/70 (85.4) |
| Patients with logMAR <1.0 in both eyes,bn (%) | 3 (5.7) | 2 (6.9) | 5 (6.1) |
| Patients ‘off chart’,cn (%) one eye/both eyes | 11 (20.8)/25 (47.2) | 3 (10.3)/13 (44.8) | 14 (17.1)/38 (46.3) |
| Patients with discordant visual acuities,dn (%) | 20 (37.7) | 10 (34.5) | 30 (36.6) |
| LogMAR: mean ± SD,e (n) | |||
| Best eye | 1.61 ± 0.64 (53) | 1.57 ± 0.61 (29) | 1.59 ± 0.62 (82) |
| Worst eye | 1.89 ± 0.49 (53) | 1.79 ± 0.44 (29) | 1.86 ± 0.47 (82) |
| Both eyes | 1.75 ± 0.58 (106) | 1.68 ± 0.54 (58) | 1.73 ± 0.57 (164) |
a n = 82 (n = 53 for idebenone; n = 29 for placebo) for all visual acuity data.
blogMAR ≥ 1.0 in both eyes corresponds to legal blindness in most countries.
clogMAR > 1.68 (patients unable to read any letter on the chart).
ddefined as patients with difference in logMAR > 0.2 between both eyes.
eapplying logMAR 2.0 for counting fingers; logMAR 2.3 for hand motion; logMAR 2.6 for light perception.
BMI = body mass index.